CAMBRIDGE, Mass. — February 12, 2026 — Leads & Copy — Agios Pharmaceuticals, Inc. (Nasdaq: AGIO), a commercial-stage biopharmaceutical company, reported its fourth quarter and full-year 2025 financial results and provided corporate updates.
The company announced PYRUKYND® (mitapivat) worldwide net revenues of $20.0 million for the fourth quarter and $54.0 million for the full year. AQVESME™ (mitapivat) for thalassemia is now available in the U.S. following FDA approval.
Agios will hold a pre-sNDA meeting with the FDA for mitapivat in sickle cell disease in the first quarter of 2026. The Phase 2 tebapivat trial in sickle cell disease is fully enrolled, with topline results expected in the second half of 2026.
As of December 31, 2025, Agios had $1.2 billion in cash, cash equivalents, and marketable securities.
According to Brian Goff, Chief Executive Officer of Agios, the U.S. launch of AQVESME is off to a strong start. The company is focused on driving a high-impact U.S. launch of AQVESME, expanding its PK activation franchise into additional high-value indications such as sickle cell disease and lower-risk myelodysplastic syndromes, and advancing its early-stage pipeline.
Mitapivat Commercial Performance
U.S. net revenue for PYRUKYND® (mitapivat 5 mg, 20 mg, 50 mg) was $16.0 million in the fourth quarter of 2025, a 49 percent increase from $10.7 million in the fourth quarter of 2024 and a 24 percent increase from $12.9 million in the third quarter of 2025. Ex-U.S. net revenue was $4.0 million in the fourth quarter of 2025.
In December 2025, the FDA approved AQVESME™ (mitapivat 100 mg) for the treatment of anemia in adults with alpha- or beta-thalassemia. AQVESME is now available in the U.S. following the implementation of its Risk Evaluation and Mitigation Strategy (REMS) program in late January 2026.
Research and Development Highlights
Topline results from the RISE UP Phase 3 trial of mitapivat in sickle cell disease were reported in November 2025.
Agios will have a pre-supplemental New Drug Application (sNDA) meeting with the FDA in the first quarter of 2026 and intends to submit a U.S. marketing application for mitapivat in sickle cell disease following that engagement. An update on the regulatory filing strategy will be provided after receipt of the meeting minutes.
Agios has completed enrollment for the Phase 2 trial of tebapivat in sickle cell disease and expects to report topline results in the second half of 2026. This trial is evaluating three tebapivat doses (2.5 mg, 5 mg, and 7.5 mg) versus placebo over a 12-week period. The primary endpoint is hemoglobin response, defined as a ≥1.0 g/dL increase in average hemoglobin concentration from Week 10 through Week 12 compared with baseline.
Fourth Quarter 2025 Financial Results
Net loss for the quarter ended December 31, 2025, was $108.0 million, compared to a net loss of $96.5 million for the quarter ended December 31, 2024.
Net product revenue from U.S. sales of PYRUKYND for the fourth quarter of 2025 was $16.0 million, compared to $10.7 million for the fourth quarter of 2024. Net product revenue from ex-U.S. sales of PYRUKYND for the fourth quarter of 2025 was $4.0 million.
Cost of sales for the fourth quarter of 2025 was $1.9 million. Research and Development (R&D) Expenses were $88.1 million for the fourth quarter of 2025, compared to $82.8 million for the fourth quarter of 2024. Selling, General and Administrative (SG&A) Expenses were $51.6 million for the fourth quarter of 2025, which were flat compared to $51.7 million for the fourth quarter of 2024.
Cash, cash equivalents, and marketable securities were $1.2 billion as of December 31, 2025, compared to $1.5 billion as of December 31, 2024.
Agios expects its cash, cash equivalents, and marketable securities, together with anticipated product revenue and interest income, will provide the financial independence to execute the U.S. commercial launch of AQVESME in thalassemia, prepare for the potential U.S. commercial launch of mitapivat in sickle cell disease, advance the company’s existing clinical programs, and opportunistically expand its pipeline through both internally and externally discovered assets.
Agios will host a conference call and live webcast today at 8:00 a.m. ET to discuss the company’s fourth quarter and full year 2025 financial results and recent business highlights. The live webcast will be accessible on the Investors section of the company’s website (www.agios.com) under the “Events & Presentations” tab. A replay of the webcast will be available on the company’s website approximately two hours after the event.
At Agios, their vision is to redefine the future of rare disease treatment. Agios is a commercial-stage biopharmaceutical company headquartered in Cambridge, Massachusetts.
Source: Agios Pharmaceuticals, Inc.
